Long-Term Prophylaxis for Hereditary Angioedema: Real-World Treatment Patterns in Selected US Allergy Clinics

Area of Science:

• Immunology
• Genetics
• Pharmacology

Background:

• Hereditary angioedema (HAE) is a rare genetic disorder causing severe swelling attacks.
• Long-term prophylactic (LTP) treatments are crucial for managing HAE attack frequency.
• Real-world data on LTP patterns in HAE patients is essential for improving care.

Purpose of the Study:

• To evaluate real-world treatment patterns of long-term prophylactic (LTP) therapies in US patients with hereditary angioedema (HAE).
• To identify reasons for discontinuation of initial LTP therapies in HAE patients.

Main Methods:

• Analysis of data from the Patient Important Outcomes National Data Repository for Hereditary Angioedema (PIONEER) registry.
• Inclusion of 179 HAE patients with C1-inhibitor deficiency (HAE-C1NH) on LTP therapy between April 2021 and March 2024.
• Observation of patients for up to three years, defining index therapy with 180 days pre- and post-initiation data.

Main Results:

• Kallikrein inhibitors were the most common index LTP (55%), followed by C1-esterase inhibitors (37%).
• 46% of patients discontinued their index LTP, with a median time to discontinuation of 12 months.
• Lack of efficacy was the primary reason for discontinuation (29%), followed by administration or dosing challenges.

Conclusions:

• Nearly half of HAE patients discontinued their initial LTP, indicating significant unmet needs in current treatment strategies.
• Efficacy, administration, and dosing challenges are key factors influencing LTP discontinuation in HAE.
• Further research with detailed data on treatment decision-making is needed to optimize HAE management.